Safety and Tolerability of RPESC-derived RPE Transplantation in Patients With Dry Age-related Macular Degeneration (AMD)

• Sponsor: Luxa Biotechnology, LLC (Industry)
• Overall Status: Recruiting
• CT.gov ID: NCT04627428
• Collaborator: National Institutes of Health (NIH) (NIH), National Eye Institute (NEI) (NIH), Regenerative Research Foundation (Other)
• Enrollment: 18
• Location: 1
• Arms: 3
• Anticipated Duration (Months): 37.8
• Patients Per Site Per Month: 0.5

Study Details

Study Description

Brief Summary

The main objective of the study is evaluation of the safety and tolerability of RPESC-RPE-4W as therapy for dry AMD.

Condition or Disease	Intervention/Treatment	Phase
Dry Age-related Macular Degeneration	Biological: RPESC-RPE-4W	Phase 1/Phase 2

Detailed Description

RPESC-RPE-4W is Allogeneic RPE stem cell (RPESC)-derived RPE cells (RPESC-RPE) isolated from the RPE layer of human cadaveric eyes are transplanted under the macular.

This first-in-human Phase 1/2a open-label dose-escalation interventional study plans to enroll a total of 18 subjects

Study Design

• Study Type: Interventional
• Anticipated Enrollment: 18 participants
• Allocation: Non-Randomized
• Intervention Model: Sequential Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Phase1/2a, Open-Label Study to Evaluate the Safety and Tolerability of RPESC-derived RPE Transplantation as Therapy for Dry Age-related Macular Degeneration (AMD)
• Actual Study Start Date: Apr 5, 2022
• Anticipated Primary Completion Date: May 31, 2025
• Anticipated Study Completion Date: May 31, 2025

Arms and Interventions

Arm	Intervention/Treatment
Experimental: 50,000 cells Six patients will receive single dose of 50,000 RPESC-RPE-4W cells in the eye.	Biological: RPESC-RPE-4W; RPESC-RPE-4W
Experimental: 150,000 cells Six patients will receive single dose of 150,000 RPESC-RPE-4W cells in the eye.	Biological: RPESC-RPE-4W; RPESC-RPE-4W
Experimental: 250,000 cells Six patients will receive single dose of 250,000 RPESC-RPE-4W cells in the eye.	Biological: RPESC-RPE-4W; RPESC-RPE-4W

Outcome Measures

Primary Outcome Measures

1. Safety and tolerability of RPESC-RPE-4W transplantation [24 months]

   The transplantation of RPESC-RPE-4W cells will be considered safe and tolerated in the absence of: Decrease in visual acuity (VA) of more than 15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters (or to worse than counting fingers at three feet) from baseline Any Grade 2 (CTCAE version 5) or greater Adverse Events (AE) related to the cell product and investigational interventions. Any evidence that the cell are contaminated with an infectious agent or serious immune response to the cell product Any evidence that the cells show tumorigenic potential

Secondary Outcome Measures

1. Change in the mean of Best Corrected Visual Acuity (BCVA) [24 months]

   Change in visual acuity will be measured by ETDRS chart.

2. Loss of ≥10 decibels of ten-degree average visual sensitivity microperimetry [24 months]

   Loss of ≥10 decibels of ten-degree average visual sensitivity will by measured by microperimetry.

3. Change in GA lesion area [24 months]

   Change in GA lesion area will be measured.

4. Evidence of structural changes [24 months]

   Structural evidence will be measured by OCT imaging, autofluorescence, fluorescein angiography, and fundus photography

Eligibility Criteria

Criteria

• Ages Eligible for Study: 55 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

1. Clinical diagnosis of dry AMD.

2. Ability to understand and give informed consent.

3. Adult male or female >55 years of age.

4. Medically suitable to undergo vitrectomy and subretinal injection (>60% on Karnofsky scale).

5. Postmenopausal if female (expected to be common for the age limitation), or the female partner of a male subject unable to father children.

6. If male, willing to use barrier and spermicidal contraception during the study.

Exclusion Criteria:

1. Allergy or hypersensitivity to dilation drops or fluorescein.

2. Active major medical conditions limiting ability to participate in the study.

3. Active malignancy or treatment with chemotherapy.

4. Systemic immunosuppressant therapy within past six months.

5. History of toxoplasmosis, retinal histoplasmosis or tuberculosis.

6. Receipt of investigational product (IP) in a clinical trial within prior six months.

7. Any other medical condition, which, in the Investigator's judgment, will interfere with the subject's ability to comply with the protocol, compromises subject safety, or interferes with the interpretation of the study results.

8. Pregnant or nursing females.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University of Michigan Kellogg Eye Center	Ann Arbor	Michigan	United States	48105

Sponsors and Collaborators

• Luxa Biotechnology, LLC
• National Institutes of Health (NIH)
• National Eye Institute (NEI)
• Regenerative Research Foundation

Investigators

• Principal Investigator: Rajesh C Rao, M.D., University of Michigan Kellogg Eye Center

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Luxa Biotechnology, LLC
• ClinicalTrials.gov Identifier: NCT04627428
• Other Study ID Numbers: RPESC-RPE-01; U01EY030581; UG3EY031810
• First Posted: Nov 13, 2020
• Last Update Posted: Jul 26, 2022
• Last Verified: Jul 1, 2022

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Macular Degeneration

Study Results

No Results Posted as of Jul 26, 2022